Pharnext, announced the opening of the first US trial site for its PLEO-CMT pivotal phase 3 clinical trial of its lead pleodrug PXT3003 in Charcot-Marie-Tooth disease type 1A (CMT1A). The clinical trial, which began enrolling patients in December 2015 in Europe, is intended to determine whether PXT3003 is effective and well tolerated in patients with CMT1A. PXT3003, developed using Pharnext's Pleotherapy R&D platform, is an oral fixed-low dose combination of (RS)-baclofen, naltrexone hydrochloride and D-sorbitol.
The study expects to enroll a total of 300 patients across 28 centres in the United States (California, Connecticut, Florida, Massachusetts, Minnesota, Missouri, New York and Washington) and in Europe (France, Germany, UK, Spain, the Netherlands and Belgium). These centres will be progressively opened and activated in 2016. The first trial site in the US that has just been activated is located in the Saint-Louis University (Missouri).
The exploratory phase 2 trial of PXT3003 demonstrated safety, tolerability and improvements beyond stabilization of CMT1A patient disability as published in the Orphanet Journal of Rare Diseases.
PLEO-CMT is a pivotal, multi-center, randomized, double blind, placebo-controlled, three-arm phase 3 study which will enroll patients aged 16 and older with mild to moderate CMT1A. Diagnosis of CMT1A will be confirmed genetically through detection of PMP22 gene duplication. Over 15 months, Pharnext will compare in parallel groups the efficacy and safety of two orally administered dosage variations of PXT3003 to placebo. Efficacy will be assessed through one primary endpoint: change in the ONLS score at 12 and 15 months of treatment to measure improvement of patients' disability with PXT3003. Additional secondary outcome measures will be assessed including functional and electrophysiological endpoints. A nine month follow-up study is planned thereafter, where all patients who will have completed the first 15 months, will receive the active PXT3003 dose.
